一是效果不如预期的好,快速通道无了,二是电话会议上说有个metavir评分药物组比安慰剂组还略略挫一点(蓝鸟上看到的)
这数据看着还行啊 有人知道到底怎么了吗?
The companies said patients receiving fazirsiran who had baseline fibrosis (n=16) showed a dose dependent mean reduction in serum mutant alpha-1 antitrypsin protein (Z-AAT) concentration at week 48 of 74%, 89%, and 94%, respectively.
All three doses led to a reduction in total liver Z-AAT with a median reduction of 94% at the postbaseline liver biopsy visit, the companies added.
The companies noted that 50% of patients achieved an improvement in fibrosis of at least one point by METAVIR stage.
Meanwhile, by week 48 patients receiving placebo who had baseline fibrosis (n=9) saw no meaningful changes from baseline in serum Z-AAT, a 26% increase in liver Z-AAT, no meaningful change in PAS-D globule burden, the companies noted.
No placebo patients experienced an improvement in portal inflammation while 44% experienced worsening, and 22% of placebo patients experienced worsening while 38% experienced an improvement in fibrosis at the postbaseline liver biopsy visit, according to the companies.
一是效果不如预期的好,快速通道无了,二是电话会议上说有个metavir评分药物组比安慰剂组还略略挫一点(蓝鸟上看到的)
对比了一下去年发的NEJM,结果-基本一致。可能是次要终点表现一般,提示治疗效果有限?
Eylea的生物类似药2025可能🇺🇸上市
我也觉得数据还行啊?最算没有超预期也不至于跌这么多啊